HDSA Research Report 2016

2016: The Momentum is Building…

Some years are less about headlines and more about steady progress towards goals. 2016 was clearly one of those years. As our work to support new therapies and curative treatments for Huntington’s disease continues, our patience continues to be tested, even as our hope builds. The breakthroughs in HD care that we seek are still on the horizon, but they are closer than ever which is why we want to reflect on all the progress and momentum that has been built in the HD research community over the past 12 months. In just this last year, three HD clinical trials – from Teva, Pfizer and Vaccinex – all completed their recruitment. The Enroll-HD registry and observational platform enrolled its 10,000th participant. Teva submitted a novel new drug application to the FDA for approval to combat the motor symptoms associated with HD. Ionis Pharmaceuticals announced that they have started administering the highest and final dose of their antisense oligonucleotide (ASO) to early stage HD patients in the first of its kind study to investigate a huntingtin lowering drug. Finally, a new study was initiated by Azevan here in the US to see if their drug, SRX-246 can improve irritability and aggression in HD patients. Research and care programs at Huntington’s Disease Society of America (HDSA) demonstrated great progress in 2016 as well. HDSA expanded their network of Centers of Excellence to 39 comprehensive care centers across 30 states. We continued to expand our patient-focused research commitments and launched a new call center to complement the HDTrialfinder and further accelerate the clinical development of new HD drugs. Since 2012, HDSA has awarded over $3 million to the HD Human Biology Project. The Human Biology Project is a critical piece of HDSA’s mission to support impactful HD research that will help guide us closer to effective therapies. The research we support is all patient-centric and done in collaboration with HD clinics from around the globe with the goal of understanding HD in the only place it occurs, in humans. In this report, you will see summaries of the nine new research projects HDSA is supporting as of November. In addition, the first HDSA Berman-Topper Family HD Career Development Fellowship, was awarded in 2016 to provide a bright young scientist with the time and resources to allow transition into an independent HD investigator. These achievements all have one thing in common. They could not have been achieved without the participation of HD families. We are indebted to the brave HD research Heroes around the world who volunteer selflessly so that we will not have to wait another generation for effective HD treatments. Thank you for being the engine that is literally powering HD science!